A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Conditions

Facioscapulohumeral Muscular Dystrophy (FSHD)

Brief summary

Change from Baseline to Week 78 in 10MWRT velocity (m/sec)

Detailed description

1. Change from Baseline to Week 78 in: o Timed-Up-and-Go (TUG) o NeuroQoL Upper Extremity Function o Quantitative Muscle Testing (QMT) total composite score (PPN), 2.1 Change from Baseline in: o 10MWRT velocity (m/sec) (excluding Week 78) o QMT total composite score (excluding Week 78) o TUG (excluding Week 78) o QMT of upper extremity composite score, lower extremity composite score, and individual muscle group scores (PPN) o Patient-reported Outcomes Measurement Information System instruments (PROMIS) (Physical Function) o PROMIS Fatigue, 2.2 Change from Baseline in: o Worst Pain Numerical Rating Scale (NRS) o Patient Global Impression of Severity(PGI-S)/Patient Global Impression of Change (PGI-C) o Quality of Life in Neurological Disorders (NeuroQoL) Upper Extremity Function (excluding Week 78) o A DUX4-regulated circulating biomarker of of FSHD disease biology o Plasma CK

Related papers

No related papers found yet.

Interventions

DRUG0.9% Saline for IV administration

DRUGAOC 1020

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Change from Baseline to Week 78 in 10MWRT velocity (m/sec)	—

Secondary

Measure	Time frame
1. Change from Baseline to Week 78 in: o Timed-Up-and-Go (TUG) o NeuroQoL Upper Extremity Function o Quantitative Muscle Testing (QMT) total composite score (PPN), 2.1 Change from Baseline in: o 10MWRT velocity (m/sec) (excluding Week 78) o QMT total composite score (excluding Week 78) o TUG (excluding Week 78) o QMT of upper extremity composite score, lower extremity composite score, and individual muscle group scores (PPN) o Patient-reported Outcomes Measurement Information System instruments (PROMIS) (Physical Function) o PROMIS Fatigue, 2.2 Change from Baseline in: o Worst Pain Numerical Rating Scale (NRS) o Patient Global Impression of Severity(PGI-S)/Patient Global Impression of Change (PGI-C) o Quality of Life in Neurological Disorders (NeuroQoL) Upper Extremity Function (excluding Week 78) o A DUX4-regulated circulating biomarker of of FSHD disease biology o Plasma CK	—

Measure

Time frame

1. Change from Baseline to Week 78 in: o Timed-Up-and-Go (TUG) o NeuroQoL Upper Extremity Function o Quantitative Muscle Testing (QMT) total composite score (PPN), 2.1 Change from Baseline in: o 10MWRT velocity (m/sec) (excluding Week 78) o QMT total composite score (excluding Week 78) o TUG (excluding Week 78) o QMT of upper extremity composite score, lower extremity composite score, and individual muscle group scores (PPN) o Patient-reported Outcomes Measurement Information System instruments (PROMIS) (Physical Function) o PROMIS Fatigue, 2.2 Change from Baseline in: o Worst Pain Numerical Rating Scale (NRS) o Patient Global Impression of Severity(PGI-S)/Patient Global Impression of Change (PGI-C) o Quality of Life in Neurological Disorders (NeuroQoL) Upper Extremity Function (excluding Week 78) o A DUX4-regulated circulating biomarker of of FSHD disease biology o Plasma CK

—

Countries

Denmark, France, Germany, Italy, Netherlands, Spain

Outcome results

None listed